Developing novel biopharmaceutical products through morphogenics

The genomics era has provided valuable information on the content of the human genome, including the structure and chromosomal location of many disease-associated loci. This wealth of information has resulted in the identification of novel targets that are amenable to biopharmaceutical product development, leading to an overall enhanced pace of therapeutic development for a broad array of disease indications. Historically, small chemical entities have been designed as therapies to gene products that are encoded by intracellular proteins, enzymes and channels. With the advent and development of biologically based (protein- and cell-based) entities (BBEs) therapies, it is now possible to create molecules that have exquisite specificity for disease targets and spare unwanted pharmacologic activity against normal tissues. In addition to the specificity, BBEs generally have lower toxicity profiles when compared with small chemical entities, making biologically-based therapeutic approaches even more attractive. One of the difficulties that has been encountered with BBEs is the ability to produce compounds with maximal pharmacologic activity as well as establishing systems that can manufacture these complex biological molecules in sufficient quantities to meet clinical demand. This review discusses a broad enabling platform technology called morphogenics that can rapidly yield robust systems that can overcome present manufacturing shortfalls as well as accelerate the development of highly efficacious BBEs from those with insufficient pharmacological activity.